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A Long Term Follow up Study in Late-onset Pompe Disease

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ClinicalTrials.gov Identifier: NCT00713245
Recruitment Status : Recruiting
First Posted : July 11, 2008
Last Update Posted : December 4, 2013
Sponsor:
Information provided by (Responsible Party):

Study Description
Brief Summary:
This study is to observe the progression of disease in late-onset Pompe disease

Condition or disease
Pompe Disease

Study Design

Study Type : Observational
Estimated Enrollment : 70 participants
Observational Model: Case-Only
Time Perspective: Prospective
Official Title: A Long Term Follow up Study in Late-onset Pompe Disease
Study Start Date : May 2008
Estimated Primary Completion Date : December 2018
Estimated Study Completion Date : December 2018


Groups and Cohorts


Outcome Measures

Primary Outcome Measures :
  1. all muscle function [ Time Frame: 10 years ]

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
individuals with acid alpha glucosidase deficiency but without obvious symptoms
Criteria

Inclusion Criteria:

  • individuals with acid alpha glucosidase deficiency without obvious symptoms
  • parents signed inform consent

Exclusion Criteria:

  • individuals with acid alpha glucosidase deficiency presenting symptoms
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00713245


Contacts
Contact: Whu-Liang Hwu, MD, PhD hwuwlntu@ntu.edu.tw

Locations
Taiwan
National Taiwan University Hospital Recruiting
Taipei, Taiwan
Contact: Yin-Hsiu Chien, MD    886-2-23123456 ext 71937      
Principal Investigator: Whu-Liang Hwu, MD, PhD         
Sub-Investigator: Yin-Hsiu Chien, MD         
Sponsors and Collaborators
National Taiwan University Hospital
Investigators
Principal Investigator: Whu-Liang Hwu, MD, PhD National Taiwan University Hospital
More Information

Responsible Party: National Taiwan University Hospital
ClinicalTrials.gov Identifier: NCT00713245     History of Changes
Other Study ID Numbers: 200804012R
First Posted: July 11, 2008    Key Record Dates
Last Update Posted: December 4, 2013
Last Verified: December 2013

Keywords provided by National Taiwan University Hospital:
Pompe disease
late onset

Additional relevant MeSH terms:
Glycogen Storage Disease Type II
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Glycogen Storage Disease
Carbohydrate Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases